Shares of Precision BioSciences, Inc. (NASDAQ:DTIL) jumped 17% on Wednesday after the company announced that its gene therapy candidate, PBGENE-DMD, received the Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA).
This special designation recognizes the urgent need for innovative treatments for Duchenne muscular dystrophy (DMD), a rare genetic condition that primarily affects boys under 18 years old, impacting about 15,000 individuals in the U.S. DMD leads to progressive muscle degeneration and weakness.
Precision’s PBGENE-DMD therapy utilizes the company’s proprietary ARCUS gene-editing platform to precisely remove exons 45 through 55 of the dystrophin gene. The company believes this strategy could help as many as 60% of DMD patients by permanently modifying DNA to restore the production of functional, near full-length dystrophin protein.
Cindy Atwell, Chief Development and Business Officer at Precision, emphasized the potential impact:
“Our technology aims to permanently rewrite a patient’s DNA to enable natural production of dystrophin protein that is functional in humans.”
With the Rare Pediatric Disease status, Precision may qualify for a Priority Review Voucher if PBGENE-DMD gains FDA approval. This voucher can be applied to expedite FDA review of another product or sold to generate non-dilutive funding.
Currently in clinical development, Precision is finalizing key toxicology studies required for investigational new drug (IND) submission and anticipates reporting initial clinical trial data in 2026.